What is gene theraby

transcript

You Are Excellent Mohamed Taha

A gene is the unit of hereditary

and carries inherited information

Amazing Facts3 billion (3,000,000,000) letters in the DNA code in every cell in your body.

There is 6 feet of DNA in each of our cells packed into a structure only 0.0004

inches across (it would easily fit on the head of a pin).

There are 100 trillion (100,000,000,000,000) cells in the body

If all the DNA in the human body was put end to end it would reach to the sun

and back over 600 times (100 trillion x 6 feet divided by 93 million miles = 1200).

If we recited the genome at one letter per second for 24 hours a day it would take

a century to recite the book of life.

The vast majority of DNA in the human genome - 97% - has no known function.

Genes who are you Exactly?

I am carried on a chromosome

I am The basic unit of heredity

Encode how to make a proteinDNARNA proteins

Proteins carry out most of life’s function.

When altered causes dysfunction of a protein

Chromosome to Gene

Genes per chromosome

Gene structure

exon 2exon 1 exon npromotor

5‘UTR

3‘UTRProtein coding sequence

exon n-1

How Function is done

Our life is maintained by molecular network systems

Molecular network system in a cell

Proteins play key roles in a living system

• Three examples of protein functions

• Catalysis:Almost all chemical reactions in a living cell are catalyzed by protein enzymes.

• Transport:Some proteins transports various substances, such as oxygen, ions, and so on.

• Information transfer:For example, hormones.

Alcoholdehydrogense oxidizes alcohols to aldehydes or ketones

Hemoglobin carries oxygen

Insulin controls the amount of sugar in the blood

TERTIARY STRUCTURE (fold)TERTIARY STRUCTURE (fold)

Genome

Expressome

Proteome

Metabolite

Functional GenomicsFrom gene to function

Dilemma

• When there is a mutation in the gene, then it will change the codon, which will change which amino acid is called for which will change the conformation of the protein which will change the function of the protein. Genetic disorders result from mutations in the genome.

Imagine

• Imagine that you accidentally broke one of your neighbor's windows.

let us imagine with each other What will you do?

Stay silent: no one will ever find out that you are guilty, but the window doesn't get fixed.

Repair it with some tape: not the best long-term solution.

Put in a new window: not only do you solve the problem, but also you do the honorable thing.

What is similar in gene therapy

I. Stay silent: ignore the genetic disorder and nothing gets fixed.

II. Try to treat the disorder with drugs or other approaches: depending on the disorder, treatment may or may not be a good long-term solution.

III. Put in a normal, functioning copy of the gene: if you can do this, it may solve the problem!

How to fix it

B C a beneficial geneA

virus modified virus

Gene therapy using an Adenovirus vector. A new gene is inserted into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein.

A virus is found which replicates by inserting its genes into the host cell's genome. This virus has three genes - A, B and C.

Gene A encodes a protein which allows this virus to insert itself

into the host's genome.

Genes B and C actually cause the disease this virus is associated with.

Replace B and C with a beneficial gene. Thus, the modified virus could introduce your 'good gene' into the host cell's genome without causing any disease.

So we use the modified virus to fix the “broken window”

Gene Therapy

Diseases

Which of the following diseases are current or potential targets of gene therapy?

Acquired

Inherited diseases

a. Cancer b. AIDS c. Diabetes d. Parkinson’s

Disease

1-one gene

Cystic fibrosis

Severe combined immunodeficiency

Adenosine deaminase and purine nucleoside phosphorylase

2- gene therapy for cancer

3-gene therapy for cardiovascular diseases

Diseases

• Acquired Inherited

DiseasesGenetic diseases:

Type 1: Single locus (gene) is defective and responsible for

the disease, 100% heritable.

: Sickle cell anemia,

Hypercholesterolemia

Cystic fibrosis

Type 2: Polygenic traits, <100% heritable, may be dependent

on environmental factors and lifestyle.

Heart disease

Cancer

Diabetes

Alcoholism

Schizophrenia

Criminal behavior

QUESTIONS

What is Gene Therapy formal definition

It is a technique for correcting defective genes that are responsible for disease development

There are several approaches:1-A normal gene inserted to compensate for a nonfunctional gene.

techniques

-An abnormal gene

changed with normal gene

An abnormal gene repaired through

selective reverse mutation

-Change the regulation of gene pairs

• Insertion of genes into individual’s cell

• Or

Tissue

To treat a disease

Hereditary disease in particular

Gene Therapy mainly aim to

• Supplement a defective mutant allele

• With a functional one

• diseases treated by gene thereby

• Can be divided to

• inherited Acquired

Diseases like

parkinsonism

Diabetes

What is gene theraby

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